ARTHEx Biotech announces ATX-01 has been granted Orphan Drug Designation by the FDA

Arthex Biotech’s lead investigationalproduct ATX-01 has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). ATX-01 is under development for the treatment of Myotonic Dystrophy Type 1 (DM1), a serious, debilitating and life-threatening disease with no known cure or currently available drug treatment.  

ATX-01 is an investigational drug with a unique, dual mechanism of action designed to address myotonic dystrophy's cause by targeting a microRNA that is key to the disease pathogenesis. ATX-01 is the first drug candidate from the Arthex Biotech proprietary platform, which provides a mechanism for enhanced delivery of the compound to tissues. Inv itro and in vivo testing support the potential for promising effects, and no rate limiting toxicity has been seen in toxicology studies in minipig and non-human primate.

DM1 is a highly disabling disease affecting more than one million people worldwide. The condition affects muscles and other tissues and most commonly manifests during adulthood, although DM1 can develop at birth in congenital form or during childhood.  Although signs and symptoms vary among affected individuals, sadly, with progression of the disease, DM1 patients experience a reduction in the ability to perform activities of daily living. At times, this can impact the ability to maintain employment and can lead to poverty and social exclusion. Moreover, patients have a significantly shortened lifespan and there is no approved treatment to slow the progression of the disease.

The FDA grants orphan drug status to products intended for the diagnosis, treatment or prevention of rare diseases and disorders that affect fewer than 200,000 people in the US. ODD provides companies with incentives that are intended to promote the development of drugs showing therapeutic promise and to address unmet medical needs.  Arthex is currently preparing to submit an IND application with the FDA.

“ATX-01 has the potential to be a viable treatment that may slow or halt the progression of DM1, relieve symptoms, and provide some relief to persons with this  devastating condition. The FDA’s orphan drug designation further underscores the potential for ATX-01 to offer hope to the DM1 patient population currently faced with limited options” said ARTHEx Biotech CEO and Founder Dr Beatriz Llamusí.  

ARTHEx Biotech is a global leader in the development of investigational anti-microRNA (antimiR) oligonucleotide compounds for unmet medical needs and difficult-to-treat diseases thanks to its proprietary ENTRY™ platform. Arthex was founded in 2019 with the support of InVivo Capital, AdBio Partners and CDTI-Innvierte as a spin-outof the University of Valencia. Arthex is currently fundraising 37 million Euros in a Series A investment round to further its drug development program.

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